Astrid Brull - National Institutes of Health

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论文共 40 篇作者统计合作学者相似作者

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Optimized Allele-Specific Silencing of the Dominant-Negative COL6A1 G293R Substitution Causing Collagen VI-related Dystrophy

Astrid Brull,Apurva Sarathy,Veronique Bolduc,Grace S. Chen,Riley M. McCarty,Carsten G. Bonnemann

Molecular Therapy — Nucleic Acids（2024）

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A humanized knock-in Col6a1 mouse recapitulates a deep-intronic splice-activating variant

Véronique Bolduc,Fady Guirguis, Berit Lubben,Lindsey Trank,Sarah Silverstein,Astrid Brull,Matthew Nalls,Jun Cheng,Lisa Garrett,Carsten G Bönnemann

bioRxiv : the preprint server for biology (2024)

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Recurring Homozygous ACTN2 Variant (p.arg506gly) Causes a Recessive Myopathy

ANNALS OF CLINICAL AND TRANSLATIONAL NEUROLOGYno. 3 (2024): 629-640

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Allele-specific CRISPR/Cas9 Editing Inactivates a Single Nucleotide Variant Associated with Collagen VI Muscular Dystrophy

Veronique Bolduc,Katherine Sizov,Astrid Brull,Eric Esposito,Grace S. Chen,Prech Uapinyoying,Apurva Sarathy,Kory R. Johnson,Carsten G. Bonnemann

Molecular Therapy — Nucleic Acidsno. 3 (2024): 102269-102269

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Use of Circular RNA Guides to Correct a Hotspot of Dominant-Negative Glycine Substitutions Causing Collagen VI-Related Dystrophies

Astrid Brull,Veronique Bolduc,Carsten G. Bonnemann

MOLECULAR THERAPYno. 4 (2023): 338-339

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Gene Therapy for Striated Muscle Laminopathy (in Vivo Study)

Mariko Okubo,Astrid Brull,Maud Beuvin,Nathalie Mougenot, Jacques Belghiti,Gisèle Bonne,Anne Bertrand

openalex（2023）

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Origin of the increased severity of LMNA-related muscular dystrophy compared with Emery-Dreyfuss muscular dystrophy

Anne Bertrand,Astrid Brull,Fériel Azibani,Ludovic Arandel,Gisèle Bonne

HAL (Le Centre pour la Communication Scientifique Directe) (2022)

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Development of gene therapy for striated muscle laminopathy

Mariko Okubo,Astrid Brull,Maud Beuvin,Gisèle Bonne,Anne Bertrand

HAL (Le Centre pour la Communication Scientifique Directe) (2022)

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Preclinical Research in McArdle Disease: A Review of Research Models and Therapeutic Strategies

Monica Villarreal-Salazar,Astrid Brull,Gisela Nogales-Gadea,Antoni L. Andreu,Miguel A. Martin,Joaquin Arenas,Alfredo Santalla,Alejandro Lucia,John Vissing,Thomas O. Krag,Tomas Pinos

GENES（2022）

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Development of Conjugated siRNAs for Allele-Specific Silencing of a Dominant Col6a3 Pathogenic Variant in Mouse Cultured Cells

Lindsey Trank,Astrid Brull,Veronique Bolduc,Annabelle Biscans,Anastasia Khvorova,Carsten G. Bonnemann

MOLECULAR THERAPY（2022）

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